Why do some cancer treating medications work and others don't?

Almost 20 years ago, a gentleman was diagnosed with metastatic melanoma, an aggressive cancer that at the time had no effective treatments. This patient was told he should measure the rest of his life in months, not years.

After the diagnosis, this patient scoured the internet for a clinical trial that could buy him more time. He eventually decided to bet his life on a Phase III trial of Canvaxin, an investigational vaccine designed to stimulate the immune system in order to fight off tumors.

Earlier clinical trials had demonstrated that the vaccine could extend the lives of people with the deadliest form of skin cancer. Unfortunately, the trial this patient was involved in, Canvaxin, failed to demonstrate that kind of survival benefit in enough people and was halted. Canvaxin was eventually destroyed.

However, a few outlier patients responded remarkably to Canvaxin before the trial was closed. This former patient is now 72 years old and cancer-free.

Until recently, such dramatic outcomes have left patients thanking some higher power for their miraculous recovery, and experts scratching their heads.

Not enough was known about cancer’s basic biology. Additionally, there was not enough technology to understand why someone fared so well on a drug that provided little to no help for most patients.

However, new genome-sequencing technologies it is increasingly possible to pinpoint genetic mutations and other molecular abnormalities that play a role in some patients’ astounding recoveries.

After studying exceptional responders, research hope to learn how and why a patient responded to a specific treatment and how to identify other patients who could benefit from the same regiment.

Decades of cancer research have shown that cancer is a remarkably diverse disease. Even cancers that begin in the same part of the body are radically different at the DNA level. For example, lung cancer is understood as not one disease, but as a variety of subtypes that each require different treatment approaches.

Cancer is increasingly defined not just by the organ in which it originated but by the mutations that drive its growth.

The American Society of Clinical Oncology’s CancerLinQ and Flatiron Health aim to collect data from millions of electronic health records. Hopefully this analysis will allow physicians to base their treatment choices on the experiences of similar patients. Other cancer institutes have created their own databases in the hopes of helping doctors match the right treatment to the right person.

To reach Gerry, call him now at 516-487-8207

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